A. McVie-Wylie

发表

Development of a simple and rapid method for producing non‐fucosylated oligomannose containing antibodies with increased effector function

Scott Estes, Qun Zhou, S. Estes, 2008, Biotechnology and bioengineering.

Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice.

N. Raben, R. Mattaliano, Seng H. Cheng, 2005, The Biochemical journal.

Tissue-specific inactivation of murine M6P/IGF2R.

R. Waterland, R. Jirtle, T. Orton, 2003, The American journal of pathology.

The influence of a polymorphism in the gene encoding angiotensin converting enzyme (ACE) on treatment outcomes in late-onset Pompe patients receiving alglucosidase alfa

Xiwen Ma, R. Pomponio, A. McVie-Wylie, 2016, Molecular genetics and metabolism reports.

Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle.

Songtao Li, D. Bali, D. Koeberl, 2011, Molecular genetics and metabolism.

Animal Model Tissue-Specific Inactivation of Murine M6P/IGF2R

R. Waterland, R. Jirtle, T. Orton, 2003 .

Inhibition of Glycogen Biosynthesis via mTORC1 Suppression as an Adjunct Therapy for Pompe Disease

K. Klinger, R. Scheule, K. Ashe, 2011 .

Long-term efficacy after [E1-, polymerase-] adenovirus-mediated transfer of human acid-alpha-glucosidase gene into glycogen storage disease type II knockout mice.

A. Amalfitano, Y. T. Chen, A. McVie-Wylie, 2001, Human gene therapy.

Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase.

N. Raben, A. Amalfitano, P. Plotz, 1999, Proceedings of the National Academy of Sciences of the United States of America.

Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial.

A. McVie-Wylie, A. McVie‐Wylie, Y. T. Chen, 2001, Genetics in medicine : official journal of the American College of Medical Genetics.

Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease.

M. O'Callaghan, N. Raben, J. McPherson, 2008, Molecular genetics and metabolism.

Transcriptional response to GAA deficiency (Pompe disease) in infantile-onset patients.

K. Klinger, S. Marie, S. Madden, 2012, Molecular genetics and metabolism.

Inhibiting TGF-β activity improves respiratory function in mdx mice.

B. Wentworth, Seng H. Cheng, Kelly A Keefe, 2011, The American journal of pathology.

Molecular cloning of a novel member of the GLUT family of transporters, SLC2a10 (GLUT10), localized on chromosome 20q13.1: a candidate gene for NIDDM susceptibility.

Yuan-Tsong Chen, A. McVie-Wylie, D. R. Lamson, 2001, Genomics.

An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease

S. Packman, O. Abdul-Rahman, P. Tanpaiboon, 2018, Genetics in Medicine.

Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis

S. Boye, S. Boye, M. Lukason, 2022, Molecular therapy. Methods & clinical development.

Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

D. Koeberl, D. K. Benjamin, Baodong Sun, 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

Multiple muscles in the AMD quail can be “cross‐corrected” of pathologic glycogen accumulation after intravenous injection of an [E1‐, polymerase‐] adenovirus vector encoding human acid‐α‐glucosidase

A. Amalfitano, T. Kikuchi, M. Mizutani, 2003, The journal of gene medicine.